Canadian clinical trial registry

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Study Description

Participants with a diagnosis of a brain tumour called medulloblastoma (categorized as high risk/very high risk or has not responded to previous treatment) will receive an oral dose of a medication called difluoromethylornithine (DFMO) twice a day while on study. 

Inclusion Criteria

• Must be 21 years old or younger to participate
• Have a confirmed diagnosis of medulloblastoma that has been categorized as high risk/very high risk or has not responded to previous treatment
• A disease assessment that includes imaging, lumbar puncture (only if previously positive) and bone marrow aspiration biopsy (only if previously positive) will be done
• Must have had enough time pass from any prior therapy (60 days from last dose of conventional chemotherapy and 45 days for high dose chemotherapy + stem cell transplantation)
• Participants must be up and about for at least 50% of waking hours
• Must meet all requirements for organ function, clinical and laboratory tests
• Females of childbearing potential must have a negative pregnancy test and must agree to use an effective birth control method.
• Participants or their legal representatives must sign an informed consent 

Additional inclusion and exclusion criteria may apply and will be discussed with you by the study team. 

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Study Description

This study is to assess the effectiveness of a drug called Difluoromethylornithine (DFMO) in combination with etoposide for patients with neuroblastoma that has come back, or is not responidng to current treatment. DFMO is a medication taken by mouth. It is an blocker of ornithine decarboxylase, an enzyme involved with polyamine biosynthesis in the cancer cells, including neuroblastoma cells. 

Inclusion Criteria

• Patients less than 31 years of age 
• Diagnosis of neuroblastoma that has come back or is not responding to treatment 
• Patients can have active or no active disease at the time of study start
• Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

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Study Description

This is a Phase 2 trial for newly diagnosed high-risk neuroblastoma to evaluate the efficacy and safety of combining the drug naxitamab with standard induction therapy. Initially, there will be 5 cycles of standard chemotherapy with Naxitamab on days 1, 3 and 5 of each cycle. Patients with an ALK mutation or amplification will have the drug ceritinib added to their treatment regimen as soon as results are available. 

Inclusion Criteria

• Subjects must have a diagnosis of neuroblastoma or ganglioneuroblastoma that meets criteria
• Subjects must be age ≤ 21 years at initial diagnosis
• Subjects must be >12 months of age at enrollment
• Subject's bloodwork must be within acceptable study ranges
• Pregnant subjects are not eligible for this study, a negative serum pregnancy test is required for female participants of childbearing potential
• Both male and female study subjects who have reached puberty must be willing to use a highly effective contraceptive method, these methods will be discussed with you by the study team
• All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines.
• Subjects enrolled on study must complete all required assessments 

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Study Description

This study is for children, teens, and young adults who have a type of bone cancer called osteosarcoma. Osteosarcoma is rare but is the most common bone cancer in young people. It happens when cancer cells make immature bone. The study includes participants whose cancer couldn’t be completely removed with surgery and who have shown some improvement or stable disease after regular chemotherapy.

In the study, participants will either:

1. Take a medication called cabozantinib (a daily tablet) along with supportive care, or
2. Receive supportive care only, which can include treatments like antibiotics, nutritional support, pain management, and other care but not cancer-specific therapy.

If the cancer worsens for those receiving only supportive care, they might switch to also taking cabozantinib if they meet certain conditions. Cabozantinib will continue as long as it is helping and well-tolerated. The study is expected to last about 24 months for each participant, but they may stay in the study longer if they are benefiting from the treatment. Participants can choose to leave the study at any time.

Inclusion Criteria

• Participants must be ≥5 and ≤30 years old
• Have a confirmed diagnosis of high-grade osteosarcoma
• Have cancer that couldn’t be fully removed after standard chemotherapy, participants must have had at least 4 cycles of chemotherapy, unless it was stopped early due to side effects.
• Recovered from most side effects of previous treatments (except for hair loss, hearing issues, or mild nerve problems).
• Be able to do most daily activities, and be up and about at least 70% of their waking hours
• Meet organs and bone marrow functioning requirements 
• Have blood pressure under control, with or without medication.
• Follow guidelines for preventing pregnancy if appropriate for their age and situation.
• Sign consent forms: participants 18 or older must sign, and younger participants need a parent or guardian’s consent, plus their agreement if required.

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team.

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Study Description

This study is to find the appropriate dose as well as to assess the safety and effectiveness of a drug called quizartinib for children with relapsed or refractory acute myeloid leukemia (AML) with a certain mutation called FLT3-ITD. Quizartinib is a drug designed to specifically block the FLT3 protein when it is abnormal in a leukemia cell.

Inclusion Criteria

• Children 1 month to 21 years of age
• Acute myeloid leukemia that has come back or is not responding to current treatment
• Presence of the FLT3-ITD mutation in the leukemia
• Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

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Study Description

This study is for people with leukemia or lymphoma that didn’t get better with treatment or came back. Doctors will take the patient’s own immune cells (T cells), change them in the lab to help them find and kill cancer, and give them back to the patient. These are called CAR T cells.

The CAR T cells in this study look for a cancer marker called CD22. 

• The first part of the study checks if the treatment is safe and finds the right dose.
• The second part checks if this treatment is effective.

Inclusion Criteria

• Participants must be 30 years or younger 
• Participants must have leukemia or lymphoma that has come back or not responded to treatment 
• Participants must be able to have blood collected or have enough T cells stored 
• Must be up and about at least 50% of waking hours
• Recovered from side effects of past treatments if using new T cells
• Must meet organ function and blood test requirements
• If participant can have children, a strong birth control must be used until 12 months after treatment 
• Participant or legal guardian must sign consent form

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team. 

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Study Description

This study is testing a new treatment for a type of cancer that grows behind the nose, called nasopharyngeal carcinoma (NPC). This study is checking if a medicine called nivolumab, when used with chemotherapy drugs such as gemcitabine and cisplatin, can help the body's immune system fight the cancer. Chemotherapy helps stop cancer from growing, and later, radiation is used to shrink the tumor.

They are also exploring whether children with NPC can safely receive less radiation, which may help reduce side effects.

Inclusion Criteria

• Participants must be ≤ 21 years of age 
• Newly diagnosed nasopharyngeal carcinoma (NPC) that has spread
• Participants must be up and about at least 60% of their waking hours
• Must meet all bloodwork and organ function requirements outlined in the study
• Participant or parent/guardian must sign informed consent form and agree to the study requirements

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team

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Study Description

 This study is eligible for STEP-1 funding. Find more information here. 

VICTORY is an early phase study for patients under 25 years who have a type of brain tumor called low grade glioma (LGG) that coms back or gets worse (recurrent or progressive). To participate on this study, the LGG must have certain genetic changes called CRAF or BRAF alterations.

The study has two parts:

Phase A: We are figuring out the best dose of a combination of two medicines, vinblastine and tovorafenib. Patients will take these medicines for a certain number of months, then just tovorafenib for a few more months. Their progress will be checked regularly with scans.

Phase B: After we find the right dose in Phase A, we'll test how well the combination of these drugs works. Patients will take these medicines for a certain number of months, then just tovorafenib for a few more months. Their progress will be checked regularly with scans.

Patients will be in the study for about two years unless their tumor gets worse (disease progression), they have bad side effects or they decide to withdrawal from the study.

Inclusion Criteria

• Patients must be less than or equal to 25 years of age at the time of enrollment
• Patient and/or their parent/guardian must sign an informed consent form to be enrolled on study
• Patients must have a diagnosis of low grade glioma (LGG) with a genetic alteration called BRAF or CRAF alteration that has come back (recurrent) or not responded to previous treatment (progressive)
• Patients must have completed at least 1 other therapy prior to enrollment on this study and recovered from its effects.
• Patients must have adequate performance status (daily activities which they are able to do) 
• Must have adequate organ function in bone marrow, kidneys, liver, heart and thyroid
• Willingness of male and female patients with reproductive potential to use double effective birth control methods, defined as one used by the patient and another by his/her partner, for the duration of treatment and for 180 days following the last dose of study drug. 
• Ability to swallow tablets or liquid, or gastric access via a nasal or gastric tube.

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team.